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The International Journal of the Royal Society of Thailand
Volume XI - 2019

Precision Medicine

in Neurological diseases

Kongkiat Kulkantrakorn 1

Abstract

Currently, a number of treatments for neurological diseases, such as
spinal muscular atrophy (SMA), are based on novel discipline known as
precision medicine. Many genetic diseases are lethal or able to cause long-
term disabilities, which not only affect the patients but also their families. In
the present article, precision medicine for certain diseases, including SMA,
hereditary transthyretin amyloidosis, Duchene muscular dystrophy, epilepsy
and migraine are reviewed.

Keywords: comprehensive genetic assessment; neurological disease; personalised

Introduction

Current medical practice is more advanced and complex than the past
decade, especially new genetic knowledge and better defined pathophysiology
of many diseases. This information leads to better prevention and treatment.
Therefore, the concept of “precision medicine” has emerged. It concerns individual
differences in terms of genetic, environment and life style. It is also known as

“personalized medicine”. Sometimes, both terms are interchangeable.
Pharmacogenetics is a part of precision medicine which involves the individual
response to medication due to the genetic basis (Precision medicine, 2019).
New medications which were marketed for the past decade focus more
on specific pathophysiology of each disease. They provide much better efficacy
and less adverse events, especially anticancer drugs (The personalized medicine
report, 2017). Regarding neurological diseases, many “untreatable” genetic

diseases have become “treatable”. These novel medications have revolutionized
the outcome of these deadly diseases. In the old days, these diseases caused

1 Faculty of Medicine, Thammasat University

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